Intellia Therapeutics was the first to prove gene editing can work in a human and now the famed CRISPR/Cas9 biotech is beefing up its ocular disease pipeline with a 10% equity stake in SparingVision.
The first-in-human results, issued in June, made Intellia the "best possible partner," said SparingVision President and CEO Stéphane Boissel in an interview with Fierce Biotech.
Intellia will provide exclusive rights to its in vivo CRISPR/Cas-9-based gene editing technology to SparingVision for up to three ocular targets. Boissel declined to disclose the specific targets, noting there are more than 250 in the eye they could go after.
“With Intellia, what we’re hoping to do is to go where the whole field should be going in terms of correcting or dealing with the consequences of mutations directly at the level of the chromosome," said Deniz Dalkara, Ph.D., chief scientific officer of SparingVision, in the joint interview. "Inherited retinal degenerations are caused by a multitude of mutations and some of these mutations are of interest to us as priority targets."
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Intellia will collect up to $200 million in biobucks per product that arises from the collaboration and also snags the option to obtain U.S. commercialization rights to products from two of the three targets. SparingVision is responsible for preclinical and clinical development for the gene editing treatments involved in the collaboration.
The companies will also research and develop self-inactivating adeno-associated virus-based vectors and lipid nanoparticle-based ways to deliver the gene editing reagents to the retina.
This isn't Intellia's first foray into the ocular arena. The company expanded a cell therapy collaboration with Novartis to include ex vivo development using ocular stem cells in December 2018. Under that $10 million cash deal, Novartis has the right to develop CRISPR/Cas9-based treatments for targets using ocular stem cells. Details on the advancement of that work have been kept under the hood.
Aside from working with Novartis on that and sickle cell disease, Intellia is also partnered with Regeneron on inherited blood disorders and the rare disease transthyretin amyloidosis. That's the disease that Intellia reported data on earlier this summer.
Back on the road
SparingVision is not wasting any time getting started on its collaboration with Intellia.
“We want to go fast and we are very soon going to elect the first target so that we start the development in 2022. Beyond that, that will be a little foolish of myself to give you a timeline," Boissel said.
Jeito Capital and 4BIO-backed SparingVision went with an equity stake rather than upfront cash "given the price tag of this kind of technology," Boissel said.
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With about €60 million ($69 million) infused into the biotech to date, SparingVision is on par to be "back on the road in the next few months to raise cash," Boissel said. The extra money will be critical to the Intellia deal, if all goes well.
Don't expect SparingVision to link up with any similar partners for a while as the biotech now has its "hands very full for the next few years," the CEO said.
The rest of SparingVision's mutation-agnostic pipeline includes a vision deterioration treatment that could land in the clinic "as soon as next year" with a GLP tox study beginning next month, Boissel said. Behind that is an asset that could enter human studies in 2024, the CEO added.